Repurposing something so that it’s new and useful is always satisfying.
If it benefits people in the process, so much the better.
In a nutshell that is what Nuformix PLC (LON:NFX) does, but in its case, it is with well-known and well-used small molecule drugs.
Utilising a technology known as cocrystallisation, Nuformix re-engineers a drug’s crystalline form by adding a molecule that improves it in a variety of ways – how well it can be absorbed by the body, for example.
Dan Gooding, co-founder and chief executive, is quick to point out it is not inventing new drugs, rather identifying something in an old product that might have a new application.
Some of these drugs have been around a long time, Gooding says, but for example only now do scientists really understand the mechanism by how it actually works, and how this might be applied to other diseases.
Drug reprofiling
Switching disease use is called drug reprofiling and means rather than starting from scratch when designing a new therapy, you take a treatment for disease A and reprofile it to treat disease B.
Because known drugs have usually been approved previously and their underlying chemistry is not changing, safety issues have been addressed, removing a major hurdle in drug development.
That leaves Nuformix just to prove that the new application passes muster against existing standard of care treatments.
“We take our cocrystal-based products through development to proof-of-concept stage before seeking to out-license to a marketing partner,” says Gooding.
Another crucial element is getting patent protection to support development towards a new application.
Reprofiling opportunities usually fail without robust IP protection, he says, as once a drug is off-patent anyone is potentially free to use it.
Trio of characteristics
Nuformix looks for three things in candidates suitable for their approach to reprofiling.
“Is the drug safe, does it have a problem our tech can improve and is there an underlying therapeutically innovative pharmacology with some level of validation in an area of unmet medical need.”
For example, Gooding says using cocrystal technology can change key drug properties, such as solubility or dissolution rate, without changing the chemistry inside.
That means a drug can dissolve faster and to a greater extent so more of the drug gets into the patient, though solubility is just one of many problems an existing drug might face.
“Many properties hold drugs back from doing other things, but in certain cases we have an opportunity to change that.
“When we discover new drug cocrystals, we can patent these new substances and leverage their improved properties in new and unique products.”
“Every drug cocrystal we discover, we breathe a new 20-year lifespan into products enabled by that substance.”
Oncology support the first product
The lead product in the portfolio currently is NXP001, a treatment for chemotherapy-induced vomiting (CINV), where clinical trials have just started.
NXP001 is targeting the large and growing oncology supportive care market, valued at £16bn in annual sales.
Existing therapies with NXP001’s mechanism work well, but because of their high price, only up to 30% of chemo patients who should receive it, do so.
The trial will demonstrate effective drug delivery using Nuformix’s simple cocrystal formula, allowing licensing partners to reduce the cost of treatment and get the product into a lot more patients.
A deal with Chinese firm Newsummit Biopharma will generate a milestone payment of £2mln, dependent on the trial results expected in May.
NXP1 to validate approach
But it’s not just NXP001 that is being tested by the trial.
Gooding wants it to validate Nuformix’s underlying cocrystal approach and has already lined up bigger targets to follow on.
Fibrosis is a catch-all phrase for scarring to all major organs in the body and stops them functioning.
In some indications such as lung and liver fibrosis, diagnosis is usually terminal.
Nuformix’s second product is a treatment for idiopathic pulmonary fibrosis (IPF), a deadly form of lung fibrosis that kills most sufferers within five years.
The two current standard treatments are blockbusters, but are highly unpleasant for patients to take and don’t greatly extend life, says Gooding.
NXP002 has already scored well against one of the standards, Esbriet, in advanced laboratory tests.
What really excites Gooding is that the mechanisms for the treatment of different forms of fibrosis are similar.
That opens the possibility of expanding NXP002 to other types of fibrosis and it is in doing this that Nuformix can potentially accrue huge value, says Gooding.
Gooding is looking at clinical trials for NXP002 towards the end of the year or early 2020, which is when it gets ‘really exciting’.
Inflexion point
“The current study for NXP001 represents the first inflexion point for every single drug in our portfolio - a clinical study to demonstrate we have a novel way of improving the delivery of a known drug.”
He adds that this part of the process costs around £700,000, which in the scheme of drug development is ‘not a lot’.
“We can achieve this first inflexion point cost effectively and with limited risk – demonstrate we have a unique position to redevelop a known drug. At this point, we will explore options to progress to patient proof-of-concept studies, hopefully for multiple assets in our portfolio.
“That’s how we can get maximum benefit for patients and investors.”
At 2.6p, Nuformix is valued at £12mln.